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Purpose

This study evaluates the efficacy and safety of investigational study drug ALK-001 in participants 8 to 45 years of age, inclusive, with symptoms and signs of autosomal recessive Stargardt disease (STGD)

Condition

Eligibility

Eligible Ages
Between 8 Years and 45 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  1. Male or female, 8 to 45 years of age (inclusive) on the day of screening. 2. Female participants of childbearing potential, as well as fertile male participants with female partners of childbearing potential, must be willing to comply with the protocol-defined methods of contraception from the time of consent until 90 days and 30 days, respectively, after the last dose of IMP. 3. Have a clinical diagnosis of typical autosomal recessive STGD macular dystrophy 4. Have provided a genetic report by any regional accredited organization that provides certified testing indicating the presence of disease-causing mutation. 5. Have signed and dated the informed consent forms (ICFs; or assent as appropriate) to participate.

Exclusion Criteria

  1. Has taken disallowed items (supplements containing vitamin A or beta-carotene, liver-based products, or prescription oral retinoid medications) within 30 days of randomization. 2. Is lactating, pregnant, or has a positive serum or urine pregnancy test at screening or at randomization, or is planning to become pregnant during the course of study. A male participant who wishes to father a child during the course of the study. 3. Has ever participated in any gene therapy, cell therapy, or device study to treat STGD, unless documented confirmation of participation in the placebo arm with no surgery is provided. Has participated in a drug study to treat STGD within the past 6 months. 4. Has participated in any drug study to treat any other condition within 5 half-lives of the investigational drug prior to screening, unless documented confirmation of participation in the placebo arm is provided. Has participated in an investigational device study within 30 days prior to screening or longer if, in the Investigator's judgment, the device could affect study outcomes. 5. Anticipates participating in any other drug or device study within the duration of the study.

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Active Comparator
Active
  • Drug: ALK-001
    Daily oral administration for 24 months
    Other names:
    • Gildeuretinol acetate
Placebo Comparator
Placebo
  • Drug: Placebo
    Daily oral administration for 24 months

Recruiting Locations

Erie Retina Research
Erie, Pennsylvania 16505
Contact:
Joseph Sala
814-422-4852
j.sala.research@protonmail.com

More Details

Status
Recruiting
Sponsor
Alkeus Pharmaceuticals, Inc.

Study Contact

For trial questions: trials@alkeuspharma.com or 877-255-7476
877-255-7476
trials@alkeuspharma.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.